TY - JOUR
T1 - Myosin-ATPase inhibitor in real-world patients with obstructive HCM: a report by the Cardiomyopathies and Pericardial Diseases WG of the Italian Society of Cardiology
AU - de Gregorio, Cesare
AU - Bellocchi, Paolo
AU - Napoli, Anna Rosa
AU - Musumeci, Beatrice
AU - Sammartino, Aniello
AU - Tini, Giacomo
AU - Todiere, Giancarlo
AU - Barison, Andrea
AU - Chubuchnyi, Vladyslav
AU - Crotti, Lia
AU - Rella, Valeria
AU - Muraru, Denisa
AU - La Maestra, Diego
AU - Campisi, Mariapaola
AU - Biagini, Elena
AU - Schiavo, Maria Alessandra
AU - Bergami, Claudio
AU - Negri, Francesco
AU - Limongelli, Giuseppe
AU - Monda, Emanuele
AU - Verrillo, Federica
AU - Vagnarelli, Fabio
AU - Lofiego, Carla
AU - Tofoni, Paolo
AU - Tomasoni, Daniela
AU - Bellicini, Maria Giulia
AU - Perugini, Enrica
AU - Dattolo, Giacomo
AU - Sguazzotti, Maurizio
AU - Mabritto, Barbara
AU - Musumeci, Giuseppe
AU - Fumero, Francesca
AU - Monte, Ines Paola
AU - Faro, Denise Cristiana
AU - Raineri, Claudia
AU - Melis, Daniele
AU - Calore, Chiara
AU - Martini, Marika
AU - Re, Federica
AU - Dei, Lorenzo-Lupo
AU - Merlo, Marco
AU - Reginato, Anna
AU - Angriman, Federico
AU - Forleo, Cinzia
AU - Guaricci, Andrea Igoren
AU - Mapelli, Massimo
AU - Patti, Giuseppe Rocco Salvatore
AU - Agostoni, Piergiuseppe
AU - Autore, Camillo
AU - Metra, Marco
AU - Canepa, Marco
AU - Castelletti, Silvia
AU - Aimo, Alberto
AU - Cappelli, Francesco
AU - Olivotto, Iacopo
AU - Sinagra, Gianfranco
AU - Imazio, Massimo
PY - 2025
Y1 - 2025
N2 - IntroductionApproximately two-thirds of patients suffering from hypertrophic cardiomyopathy present with an obstructive (HOCM) physiology. For years, medical therapy has been limited to beta blockers, verapamil and/or disopyramide. Recently, a novel class of drugs, the allosteric inhibitors of the cardiac-specific myosin head adenosine triphosphatase (ATPase), have been demonstrated to be effective in relieving the dynamic obstruction and related clinical condition. In July 2024 the Cardiomyopathies and Pericardial Diseases WG of the Italian Society of Cardiology started with a nationwide multicentre registry aimed at investigating the pathophysiology of dynamic obstruction in real-world patients with HOCM. Based on the medical records, this brief report deals with the proportion of patients who were eligible for Mavacamten based on the Explorer-HCM entry criteria, and then admitted for compassionate use by the end of 2024.Methods and resultsThe Hypertrophic Obstructive Physiology Study (HOPS) was designed as a registry on consecutive adult patients admitted to 19 tertiary Cardiac Centres in Italy until June 2024. A total of 424 patients, 53% males, aged 64 ± 13 years, were included. We retrospectively recognized 200 Mavacamten-eligible patients (47.2%) on 5 Explorer-HCM requirements. Forty out of this latter group, along with 15 more patients on 4 criteria, were admitted to the compassionate use programme (n = 55, 13% of the whole population). Forty-three showed subaortic obstruction and 12 a mid-ventricular variant. Ethical committee approval items varied among centres and regions.DiscussionThis study confirmed our recent demonstration that approximately half of real-world HOCM patients are suitable for Mavacamten therapy based on the full Explorer-HCM trial entry criteria. Due to the current limitation of compassionate use programmes in Italy, only one in four patients was admitted for treatment.
AB - IntroductionApproximately two-thirds of patients suffering from hypertrophic cardiomyopathy present with an obstructive (HOCM) physiology. For years, medical therapy has been limited to beta blockers, verapamil and/or disopyramide. Recently, a novel class of drugs, the allosteric inhibitors of the cardiac-specific myosin head adenosine triphosphatase (ATPase), have been demonstrated to be effective in relieving the dynamic obstruction and related clinical condition. In July 2024 the Cardiomyopathies and Pericardial Diseases WG of the Italian Society of Cardiology started with a nationwide multicentre registry aimed at investigating the pathophysiology of dynamic obstruction in real-world patients with HOCM. Based on the medical records, this brief report deals with the proportion of patients who were eligible for Mavacamten based on the Explorer-HCM entry criteria, and then admitted for compassionate use by the end of 2024.Methods and resultsThe Hypertrophic Obstructive Physiology Study (HOPS) was designed as a registry on consecutive adult patients admitted to 19 tertiary Cardiac Centres in Italy until June 2024. A total of 424 patients, 53% males, aged 64 ± 13 years, were included. We retrospectively recognized 200 Mavacamten-eligible patients (47.2%) on 5 Explorer-HCM requirements. Forty out of this latter group, along with 15 more patients on 4 criteria, were admitted to the compassionate use programme (n = 55, 13% of the whole population). Forty-three showed subaortic obstruction and 12 a mid-ventricular variant. Ethical committee approval items varied among centres and regions.DiscussionThis study confirmed our recent demonstration that approximately half of real-world HOCM patients are suitable for Mavacamten therapy based on the full Explorer-HCM trial entry criteria. Due to the current limitation of compassionate use programmes in Italy, only one in four patients was admitted for treatment.
KW - heart failure
KW - hypertrophic cardiomyopathy
KW - myosin ATP-ase inhibitors
KW - obstructive physiology
KW - treatment
KW - heart failure
KW - hypertrophic cardiomyopathy
KW - myosin ATP-ase inhibitors
KW - obstructive physiology
KW - treatment
UR - https://iris.uniupo.it/handle/11579/222466
U2 - 10.2459/jcm.0000000000001746
DO - 10.2459/jcm.0000000000001746
M3 - Article
SN - 1558-2027
VL - 26
SP - 381
EP - 385
JO - Journal of Cardiovascular Medicine
JF - Journal of Cardiovascular Medicine
IS - 7
ER -